MedPath

Efficacy, Safety, and Tolerability of AVP-786 for the Treatment of Agitation in Participants With Dementia of the Alzheimer's Type

Phase 3
Completed
Conditions
Agitation in Participants With Dementia of the Alzheimer's Type
Interventions
Drug: Placebo
Drug: AVP-786-18
Drug: AVP-786-28
Drug: AVP-786-42.63
Registration Number
NCT02442778
Lead Sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Brief Summary

Participants with agitation secondary to dementia of the Alzheimer's type. The diagnosis of probable Alzheimer's disease (AD) will be based on the "2011 Diagnostic Guidelines for Alzheimer's Disease" issued by the National Institute on Aging (NIA)-Alzheimer's Association (AA) workgroups.

Detailed Description

Eligible participants for this study must have a diagnosis of probable AD and must have clinically meaningful agitation secondary to AD.

This is a multicenter, randomized, placebo-controlled study, consisting of 12 weeks of treatment.

Approximately 470 participants will be enrolled at approximately 75 centers in North America.

Study medication will be administered orally twice-daily from Day 1 through Week 12 (Day 85). Screening will occur within approximately 4 weeks prior to randomization. Following screening procedures for assessment of inclusion and exclusion criteria, eligible participants will be randomized into the study.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
522
Inclusion Criteria
  • Diagnosis of probable Alzheimer's Disease (AD) according to the 2011 National Institute on Aging-Alzheimer's Association (NIA-AA) working groups criteria
  • The participant has clinically significant, moderate/severe agitation at the time of screening and for at least 2 weeks prior to randomization
  • The diagnosis of agitation must meet the International Psychogeriatric Association (IPA) provisional definition of agitation
  • Either out participants or residents of an assisted-living facility or a skilled nursing home
  • Clinical Global Impression of Severity of Illness (CGIS) score assessing Agitation is >=4 (moderately ill) at screening and baseline
  • Mini-Mental State Examination (MMSE) score is between 6 and 26 (inclusive) at screening and baseline
  • Caregiver who is able and willing to comply with all required study procedures. In order to qualify as a reliable informant (i.e., caregiver) capable of assessing changes in participant's condition during the study, the individual must spend a minimum of 2 hours per day for 4 days per week with the participant.
Exclusion Criteria
  • Participant has dementia predominantly of non-Alzheimer's type (e.g., vascular dementia, frontotemporal dementia, Parkinson's disease, substance-induced dementia)
  • Participants with co-existent clinically significant or unstable systemic diseases that could confound the interpretation of the safety results of the study (e.g., malignancy, poorly controlled diabetes, poorly controlled hypertension, unstable pulmonary, renal or hepatic disease, unstable ischemic cardiac disease, dilated cardiomyopathy, or unstable valvular heart disease)
  • Participant with myasthenia gravis

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants were administered AVP-786 matching placebo capsules, orally, twice daily (BID) for up to 12 weeks.
AVP-786-28AVP-786-18Participants were administered AVP-786-18 capsule, orally, once daily (QD) along with AVP-786 matching placebo capsule, orally, QD during Week 1 followed by AVP-786-18 capsules, orally, BID during Weeks 2, 3 and AVP-786-28 capsules, orally, BID during Weeks 4 to 12.
AVP-786-28AVP-786-28Participants were administered AVP-786-18 capsule, orally, once daily (QD) along with AVP-786 matching placebo capsule, orally, QD during Week 1 followed by AVP-786-18 capsules, orally, BID during Weeks 2, 3 and AVP-786-28 capsules, orally, BID during Weeks 4 to 12.
AVP-786-42.63AVP-786-28Participants were administered AVP-786-28 capsules, orally, QD along with AVP-786 matching placebo capsule, orally, QD during Week 1 followed by AVP-786-28 capsules, orally, BID during Weeks 2, 3, and AVP-786-42.63 capsules, orally, BID during Weeks 4 to 12.
AVP-786-42.63AVP-786-42.63Participants were administered AVP-786-28 capsules, orally, QD along with AVP-786 matching placebo capsule, orally, QD during Week 1 followed by AVP-786-28 capsules, orally, BID during Weeks 2, 3, and AVP-786-42.63 capsules, orally, BID during Weeks 4 to 12.
Primary Outcome Measures
NameTimeMethod
Change From Baseline to Week 12 in the Cohen-Mansfield Agitation Inventory (CMAI) Composite ScoreBaseline, Week 12

The CMAI was used to assess the frequency of manifestations of agitated behaviors in elderly participants. It consists of 29 agitated items rated on a 7-point scale of frequency: 1, never; 2, less than once a week; 3, once or twice a week; 4, several times a week; 5, once or twice a day; 6, several times a day; 7, several times an hour. The CMAI total score ranges from 29 to 203. Higher scores indicate worsening of the condition. Negative change from baseline indicates improvement. Mixed Model Repeated Measures (MMRM) was used for the analysis.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline to Week 12 in the NPI Agitation/Aggression Caregiver Distress ScoreBaseline, Week 12

The NPI is a retrospective interview covering 12 neuropsychiatric symptom domains and is used to evaluate caregiver distress. Each NPI domain is rated by the caregiver for symptom frequency and severity. Symptom frequency is rated as:1, occasionally; 2, often; 3, frequently; 4, very frequently. Symptom severity is rated as:1, mild; 2, moderate; 3, marked severe. The total caregiver distress score is calculated as the frequency score multiplied by the severity score and thus ranges from 1 to 12 with a higher score indicating worsening of symptoms. Negative change from baseline indicates improvement in symptoms. MMRM was used for the analysis.

Change From Baseline to Week 12 in the NPI Aberrant Motor Behavior Domain ScoreBaseline, Week 12

The NPI is a retrospective interview covering 12 neuropsychiatric symptom domains and is used to evaluate aberrant motor behavior. Each NPI domain is rated by the caregiver for symptom frequency and severity. Symptom frequency is rated as: 1, occasionally; 2, often; 3, frequently; 4, very frequently. Symptom severity is rated as: 1, mild; 2, moderate; 3, marked severe. The total domain score is calculated as the frequency score multiplied by the severity score and thus ranges from 1 to 12 with a higher score indicating worsening of symptoms. Negative change from baseline indicates improvement in symptoms. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Clinical Global Impression of Severity of Illness (CGIS)-Agitation Domain ScoreBaseline, Week 12

The CGIS-Agitation is an observer-rated scale that measures illness severity. The CGIS-Agitation is a 7-point (1-7) scale (1=normal, not at all ill participants; 7=among the most extremely ill participants) and is assessed for severity of agitation. A value of 0 is given to participants who are not assessed. Higher scores indicate poor health of participants. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Dementia Quality of Life (DEMQOL) ScoreBaseline, Week 12

The DEMQOL scale is used to evaluate health-related QOL in participants with dementia and their caregivers. There are 2 versions of the DEMQOL: a 28-item version (rated by the participant); and a 31-item version (DEMQOL-proxy, rated by the caregiver). Both versions are recommended for evaluating participants (and their caregivers) with mild to moderate dementia. The DEMQOL total score ranges from 28 to 112. The DEMQOL-proxy is used for participants with severe dementia; the total score ranges from 31 to 124. For both versions, higher scores indicate greater QOL. MMRM was used for the analysis.

Number of Participants With the Change From Baseline in the General Medical Health Rating (GMHR) Score at Week 12Week 12

The GMHR is a global clinical rating for medical health, designed to quantify in a single number (1 to 4) the severity of general comorbidity in a participant with dementia. The ratings are: 1 = poor; 2 = fair; 3 = good; 4 = excellent to very good. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Zarit Burden Interview (ZBI) ScoreBaseline, Week 12

The ZBI is a 22-item scale used to assess the impact of a participants' disabilities on the caregiver's life. It is designed to reflect the burden experienced by caregivers of dementia participants and can either be completed by the caregiver or administered as an interview. Each item of the scale is rated to reflect the burden using the 5-point scale: 0=Never; 1=Rarely; 2=Sometimes; 3=Quite Frequently; 4=Nearly Always. The ZBI is scored by summing the responses of the individual questions and ranges from 0 to 88. Higher scores indicate greater caregiver distress. Negative change from baseline indicates less distress. MMRM was used for the analysis.

Change From Baseline to Week 12 in the NPI Irritability/Lability Domain ScoreBaseline, Week 12

The NPI is a retrospective interview covering 12 neuropsychiatric symptom domains, including the irritability/lability domain score. Each NPI domain is rated by the caregiver for symptom frequency and severity. Symptom frequency is rated as:1, occasionally; 2, often; 3, frequently; 4, very frequently. Symptom severity is rated as:1, mild; 2, moderate; 3, severe. The total domain score is calculated as the frequency score multiplied by the severity score and thus ranges from 1 to 12. Higher scores indicate worsening of the symptoms. Negative change from baseline indicates improvement in symptoms. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) ScoreBaseline, Week 12

The ADAS is designed to evaluate the cognitive and non-cognitive behavioral dysfunction characteristics of participants with AD. The cognitive subscale (ADAS-cog) consists of 11 subsets related to memory, praxis, and language. ADAS-cog scores range from 0 to 70. Higher scores indicate greater cognitive impairment. Negative change from baseline indicates less cognitive impairment. MMRM method was used for analysis.

Change From Baseline to Week 12 in the Alzheimer's Disease Cooperative Study-Clinical Global Impression of Change (ADCS-CGIC) Overall RatingBaseline, Week 12

The ADCS-CGIC rating scale provides a reliable means to assess change from a Baseline level of global function within the time frame of the trial. ADCS-CGIC-Overall focuses on the clinician's observations of change in the participant's cognitive, functional, and behavioral performance. The ADCS-CGIC-Overall responses (1-7) are rated as: 1 = marked improvement, 2 = moderate improvement, 3 = minimal improvement, 4 = no change, 5 = minimal worsening, 6 = moderate worsening, or 7 = marked worsening. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Neuropsychiatric Inventory (NPI) Agitation/Aggression Domain ScoreBaseline, Week 12

The NPI is a retrospective caregiver-informant interview covering 12 neuropsychiatric symptom domains. The Agitation/Aggression domain is designed to collect information on the behavioral aspects of agitation/aggression in participants with probable AD and clinically meaningful agitation secondary to AD. Each NPI domain is rated by the caregiver for symptom frequency and severity. Symptom frequency is rated as:1, occasionally; 2, often; 3, frequently; 4, very frequently. Symptom severity is rated as: 1, mild; 2, moderate; 3, severe. The total domain score is calculated as the frequency score multiplied by the severity score and thus ranges from 1 to 12. Higher scores indicate worsening symptoms. Negative change from baseline indicates improvement in symptoms. MMRM was used for the analysis.

Change From Baseline to Week 12 in the Patient Global Impression of Change (PGIC) ScoreBaseline, Week 12

The PGIC is a 7-point (1-7) scale used to assess treatment response: 1 = very much improved, = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, or 7 = very much worse. Higher scores indicate less response to treatment. MMRM was used for the analysis.

Change From Baseline in the Cornell Scale for Depression in Dementia (CSDD) ScoreBaseline, Week 12

The CSDD scale is used to assess signs/symptoms of major depression in participants with dementia. CSDD has 19 items, and each item is rated for severity on the following scale of 0 to 2 (0 =absent, 1= mild/intermittent 2=severe). CSDD score is calculated by summing non-missing scores from each item score. The scale ranges from 0 (no depression) to 38 (maximum depression). Scores above 10 indicate a probable major depression, above 18 indicate a definite major depression, and below 6 as a rule are associated with the absence of significant depressive symptoms. Higher score indicated maximum depression. MMRM was used for the analysis.

Relative Change From Baseline to Week 12 in the Modified Alzheimer's Disease Cooperative Study-Clinical Global Impression of Change (mADCS-CGIC)-Agitation ScoreBaseline, Week 12

The mADCS-CGIC-Agitation is a modified version of the ADCS-CGIC containing additional questions related to agitation and an assessment of the Clinician's Impression of Change focused specifically on agitation. Participants are asked to rate their impression of change as: 1=Marked Improvement; 2=Moderate Improvement; 3=Minimal Improvement; 4=No Change; 5=Minimal Worsening; 6=Moderate Worsening; 7=Marked Worsening. Higher scores indicate worsening of agitation and positive change from baseline indicates worsening. MMRM was used for the analysis.

Change From Baseline to Week 12 in the NPI Total ScoreBaseline, Week 12

NPI evaluates both frequency and severity of 12 neuropsychiatric disturbances including delusions, hallucinations, agitation/aggression, depression/dysphoria, anxiety, elation/euphoria, apathy/indifference, disinhibition, irritability/lability, motor disturbance, night time behaviors, as well as appetite/eating. Total domain score= frequency x severity and thus ranges from 1 to 12. NPI domain is rated by caregiver for symptom frequency and severity. Frequency is rated as:1=occasionally, 2=often, 3= frequently, and 4=very frequently. Severity is rated as:1=mild,2=moderate,3=severe. Frequency and severity rating scales has defined anchor points to enhance reliability of caregiver responses. Caregiver distress is rated for each positive neuropsychiatric symptom using following anchored scores. It is rated as 0=not at all,1=minimal,2=mild,3=moderate,4=severe,5=very severe. Individual Item scores are added to yield a possible total score of 0 to 144. MMRM was used for the analysis.

Resource Utilization in Dementia (RUD) Score: Number of Hours Per Day the Caregiver Spent Assisting the ParticipantWeek 12

The RUD evaluates dementia participants' utilization of formal and informal healthcare resources, including hospitalizations and doctor visits, living assistance, and time spent by nonprofessional caregivers. Information on hours per day the caregiver spent assisting participant were reported in this outcome measure, using the following questions:

Q1= On a typical care day during the last 30 days, how much time per day did you assist the participant with tasks such as toilet visits, eating, dressing, grooming, walking and bathing? Q2= On a typical care day during the last 30 days, how much time per day did you assist the participant with tasks such as shopping, food preparation, housekeeping, laundry, transportation, taking medication and managing financial matters? Q3= On a typical care day during the last 30 days, how much time per day did you spend supervising (that is, preventing dangerous events) the participant?

Resource Utilization in Dementia (RUD) Score: Number of Days the Caregiver Spent Assisting the ParticipantWeek 12

The RUD evaluates dementia participants' utilization of formal and informal healthcare resources, including hospitalizations, doctor visits, living assistance, and time spent by nonprofessional caregivers. Information on days the caregiver spent assisting participant were reported in this outcome measure, using the following questions:

Q1= During the last 30 days, how many days did you spend providing these (toilet visits, eating, dressing, grooming, walking and bathing) services to the participant? Q2= During the last 30 days, how many days did you spend providing these (shopping, food preparation, housekeeping, laundry, transportation, taking medication and managing financial matters) services to the participant? Q3= During the last 30 days, how many days did you spend providing these services (supervising) to the participant?

Resource Utilization in Dementia (RUD) Score: Number of Visits to Hospital, Emergency, and Healthcare ProfessionalWeek 12

The RUD evaluates dementia participants' utilization of formal and informal healthcare resources, including hospitalizations, doctor visits, living assistance, and time spent by nonprofessional caregivers. Information on the number of hospital visits, emergency visits and visits to healthcare professional were reported in this outcome measure using the following questions:

Q1= During the last 30 days, how many times did the participant receive care in a hospital emergency room (for less than 24 hours)? Q2= During the last 30 days, how many times did the caregiver receive care in a hospital emergency room (for less than 24 hours)? Q3= During the last 30 days total number of visits by participant to a health care professional? Q4= During the last 30 days, how many times (number of visits for each) the participant visited any other health care professional?

Trial Locations

Locations (81)

Texas Neurology, P.A.

🇺🇸

Dallas, Texas, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

Houston Methodist Neurological Institute

🇺🇸

Houston, Texas, United States

Clinical Trial Network

🇺🇸

Houston, Texas, United States

Premier Clinical Research Institute, Inc.

🇺🇸

Miami, Florida, United States

BioMed Research Institute

🇺🇸

Miami, Florida, United States

Coral Research Clinic Corp

🇺🇸

Miami, Florida, United States

CCM Clinical Research Group

🇺🇸

Miami, Florida, United States

AMB Research Center, Inc.

🇺🇸

Miami, Florida, United States

CTI Clinical Research Center

🇺🇸

Cincinnati, Ohio, United States

BG Neurology

🇺🇸

Spartanburg, South Carolina, United States

Veteran Affairs Medical Center, Salem Virginia

🇺🇸

Salem, Virginia, United States

Pharmaceuticals Research Associates, Inc.

🇺🇸

Salt Lake City, Utah, United States

JEM Research Institute

🇺🇸

Atlantis, Florida, United States

Brain and Spine Center

🇺🇸

Chandler, Arizona, United States

Health Initiatives Research

🇺🇸

Fayetteville, Arkansas, United States

Behavioral Research Specialists, LLC

🇺🇸

Glendale, California, United States

California Neurological Services

🇺🇸

Panorama City, California, United States

Havana Research Institute

🇺🇸

Pasadena, California, United States

Pacific Research Network, Inc

🇺🇸

San Diego, California, United States

Viking Clinical Research

🇺🇸

Temecula, California, United States

Syrentis Clinical Research

🇺🇸

Santa Ana, California, United States

Clinical Research Of Brandon, LLC

🇺🇸

Brandon, Florida, United States

Moonshine Research Center, Inc

🇺🇸

Doral, Florida, United States

Science Connections, LLC

🇺🇸

Doral, Florida, United States

Finlay Medical Research Corp

🇺🇸

Greenacres City, Florida, United States

Indago Research & Health Center, Inc.

🇺🇸

Hialeah, Florida, United States

Reliable Clinical Research,LLC

🇺🇸

Hialeah, Florida, United States

New Life Medical Research Center, Inc.

🇺🇸

Hialeah, Florida, United States

Maxblue Institute

🇺🇸

Hialeah, Florida, United States

Research in Miami, Inc

🇺🇸

Hialeah, Florida, United States

The Research Center, Inc

🇺🇸

Hialeah, Florida, United States

Clinical Neuroscience Solutions, Inc.

🇺🇸

Jacksonville, Florida, United States

Alzheimer's Research and Treatment Center

🇺🇸

Lake Worth, Florida, United States

Project 4 Research

🇺🇸

Miami, Florida, United States

United Health Research Corp

🇺🇸

Miami, Florida, United States

DADE Research Center, LLC

🇺🇸

Miami, Florida, United States

Advance Medical Research Center

🇺🇸

Miami, Florida, United States

The Neurology Research Group, LLC

🇺🇸

Miami, Florida, United States

P&S Reasearch

🇺🇸

Miami, Florida, United States

Kendall Research Institute

🇺🇸

Miami, Florida, United States

Nuovida Research Center Corp.

🇺🇸

Miami, Florida, United States

Collier Neurologic Specialists, LLC

🇺🇸

Naples, Florida, United States

Lazlo J. Mate, MD, PA

🇺🇸

North Palm Beach, Florida, United States

IMIC Inc.

🇺🇸

Palmetto Bay, Florida, United States

University of West Florida

🇺🇸

Pensacola, Florida, United States

Neurology Research Institute Palm Beach, LLC

🇺🇸

West Palm Beach, Florida, United States

Emory Brain Health Center

🇺🇸

Atlanta, Georgia, United States

Columbus Research & Wellness Institute, INC

🇺🇸

Columbus, Georgia, United States

Josephson Wallack Munshower Neurology, PC

🇺🇸

Indianapolis, Indiana, United States

Mir Neurology

🇺🇸

Hagerstown, Maryland, United States

The Samuel & Alexia Bratton Memory Clinic

🇺🇸

Easton, Maryland, United States

Baptist Health Medical Group

🇺🇸

Richmond, Kentucky, United States

Boston Center for Memory

🇺🇸

Newton, Massachusetts, United States

AMAC Research Institute

🇺🇸

North Dartmouth, Massachusetts, United States

Michigan State University

🇺🇸

East Lansing, Michigan, United States

Center for Emotional Fitness

🇺🇸

Cherry Hill, New Jersey, United States

Eastside Comprehensive Medical Center, LLC

🇺🇸

New York, New York, United States

Brooklyn Medical Institute

🇺🇸

Brooklyn, New York, United States

Integrative Clinical Trials, LLC

🇺🇸

Brooklyn, New York, United States

ANI Neurology PLLC dba Alzheimer's Memory Center

🇺🇸

Charlotte, North Carolina, United States

Daystar Clinical Research Inc

🇺🇸

Akron, Ohio, United States

Valley Medical Research

🇺🇸

Centerville, Ohio, United States

Heritage Valley Medical Group, Inc.

🇺🇸

Beaver, Pennsylvania, United States

The Birches at Newton / Family Medical Associates

🇺🇸

Levittown, Pennsylvania, United States

Cleveland Clinic Lou Ruvo Center for Brain Health at Lakewood Hospital

🇺🇸

Lakewood, Ohio, United States

IPC Research

🇺🇸

Waukesha, Wisconsin, United States

The Medical Art Health Research Group

🇨🇦

West Vancouver, Bristish Columbia, Canada

Texas Medical Research Associates, L.L.C.

🇺🇸

San Antonio, Texas, United States

Meridien Research

🇺🇸

Brooksville, Florida, United States

Irvine Center for Clinical Research

🇺🇸

Irvine, California, United States

Territory Neurology & Research Institute

🇺🇸

Tucson, Arizona, United States

The NeuroCognitive Institute

🇺🇸

Mount Arlington, New Jersey, United States

Clinical Neuroscience Research Associates, Inc. dba The Memory Clinic

🇺🇸

Bennington, Vermont, United States

Burke Rehabilitation Hospital

🇺🇸

White Plains, New York, United States

Denver Neurological Research, LLC

🇺🇸

Denver, Colorado, United States

Compass Research, LLC

🇺🇸

Orlando, Florida, United States

Herbert Harris, MD, PhD, PA

🇺🇸

Chapel Hill, North Carolina, United States

RH Johnson VA Medical Center

🇺🇸

Charleston, South Carolina, United States

Dr. Alexander McIntyre Inc.

🇨🇦

Calgary, Alberta, Canada

Thomas Jefferson University

🇺🇸

Philadelphia, Pennsylvania, United States

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